A clinical trial evaluates the utility of genetically altered autologous hematopoietic stem cells for the treatment of childhood cerebral adrenoleukodystrophy, a deadly disease portrayed in the movie Lorenzo’s oil. The interim results are encouraging.

In 1984, at the age of six, Lorenzo Odone was a happy, bright, precocious kid, who was fluent in English, French and Italian and loved Greek mythology. However, suddenly that year he appeared to become hard of hearing, started to stumble a lot, and displayed mood swings. Concerned, his parents took him to a doctor who quickly ordered a brain scan. The result was devastating – Lorenzo was diagnosed with childhood cerebral adrenoleukodystrophy (CCALD), a disease that is rare, fatal, and untreatable. Doctors gave him two more years to live. The courageous struggle of his parents to find a cure was portrayed in the 1992 movie Lorenzo’s oil. The real life Lorenzo beat the odds by remaining alive until 2008. However, his disease never relented.

A new report shows that autologous therapy with bone marrow-derived mesenchymal stem cells secreting neurotrophic factors may slow progression of amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s Disease).

Henry Louis (“Lou”) Gehrig had a stellar career as first baseman for the New York Yankees. In 17 seasons, through the 1920’s and 1930’s, he hit almost 500 home runs. He seemed indefatigable and powerful, nicknamed “The Iron Horse”. That is, until, in the second half of the 1938 season, his abilities sharply deteriorated. In June 1939, he was diagnosed with amyotrophic lateral sclerosis (ALS). Lou Gehrig died two years later.